Article
Cystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies.
Expert opinion on investigational drugs - 1 Mar 2011
Antoniu Sabina Antonela
Abstract excerpt
INTRODUCTION: Several types of mutations in the cystic fibrosis transmembrane regulator (CFTR) gene lead to abnormal CFTR protein and alterations of chloride and sodium transmembrane transportation in cystic fibrosis (CF). Some investigational compounds such as VX-770 can improve CFTR protein function. AREAS COVERED: This paper discusses the results of a Phase II study investigating the safety and efficacy of...
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